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| ID | Type | Description | Link |
|---|---|---|---|
| 2018-000126-55 | EudraCT Number |
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The purpose of this study is to evaluate safety and tolerability of VX-121 in healthy subjects and in subjects with cystic fibrosis (CF).
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Part A: Pooled Placebo (Cohorts A1-5; Except A3) | Placebo Comparator | Participants received single dose of placebo matched to VX-121. |
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| Part A: VX-121 (Cohort A1) | Experimental | Participants received single dose of VX-121 10 milligrams (mg). |
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| Part A: VX-121 (Cohort A2) | Experimental | Participants received single dose of VX-121 20 mg. |
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| Part A: VX-121 (Cohort A3) | Experimental | Participants received single dose of VX-121 5 mg or matched placebo without milk, followed by open label VX-121 5 mg with milk. |
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| Part A: VX-121 (Cohort A4) | Experimental | Participants received single dose of VX-121 40 mg. |
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| Part A: VX-121 (Cohort A5) |
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Placebo (matched to VX-121 suspension) | Drug | Placebo matched to VX-121 suspension for oral administration. |
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| Measure | Description | Time Frame |
|---|---|---|
| Safety and Tolerability as Assessed by Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) | From Day 1 Through Safety Follow-up (up to Day 15 for Part A [except Cohorts A3 and A9], up to Day 26 for Cohort A3, up to Day 34 for Cohort A9, up to Day 20 for Part B, up to Day 24 for Part C and up to Week 9 for Part D) |
| Measure | Description | Time Frame |
|---|---|---|
| Part A: Maximum Observed Concentration (Cmax) of VX-121 | Cohorts A1-5 (Except A3): Pre-dose up to 240 hours post-dose; Cohorts A3 and A9: Pre-dose up to 168 hours post-dose | |
| Part A: Area Under the Concentration Versus Time Curve From the Time of Dosing to the Last Measurable Concentration (AUC[0-last]) of VX-121 |
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Key Inclusion Criteria:
Part A, B, and C: Healthy Volunteers
Part D: Subjects with CF
Key Exclusion Criteria:
Part A, B and C: Healthy Volunteers
Part D: Subjects with CF
Other protocol defined Inclusion/Exclusion criteria may apply
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Academic Medical Center | Amsterdam | Netherlands | ||||
| PRA Health Sciences Onderzoekscentrum UMCG |
This study included 4 parts: Parts A, B, and C were conducted in healthy adult participants; Part D was conducted in adult cystic fibrosis (CF) participants.
A total of 115 unique participants enrolled in this study.
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| ID | Title | Description |
|---|---|---|
| FG000 | Part A: Pooled Placebo (Cohorts A1-5; Except A3) | Participants received single dose of placebo matched to VX-121. |
| FG001 | Part A: VX-121 (Cohort A1) | Participants received single dose of VX-121 10 milligrams (mg). |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Part A |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Jul 27, 2018 | Apr 29, 2022 |
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| Experimental |
Participants received single dose of VX-121 60 mg. |
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| Part A: VX-121 (Cohort A9) | Experimental | Participants received single dose of VX-121 10 mg suspension on Day 1, VX-121 10 mg tablet on Day 9, followed by VX-121 10 mg tablet with milk on Day 17. |
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| Part B: Pooled Placebo (Cohorts B1-4) | Placebo Comparator | Participants received placebo matched to VX-121 for 10 days. |
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| Part B: VX-121 (Cohort B1) | Experimental | Participants received VX-121 10 mg once daily (qd) for 10 days. |
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| Part B: VX-121 (Cohort B2) | Experimental | Participants received VX-121 20 mg qd for 10 days. |
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| Part B: VX-121 (Cohort B3) | Experimental | Participants received VX-121 40 mg qd for 10 days. |
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| Part B: VX-121 (Cohort B4) | Experimental | Participants received VX-121 60 mg qd for 10 days. |
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| Part C: Pooled Placebo (Cohorts C1-3) | Placebo Comparator | Participants received placebo matched to VX-121/TEZ/IVA for 14 days. |
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| Part C: VX-121 (Cohort C1) | Experimental | Participants received VX-121 10 mg qd/TEZ 100 mg qd/IVA 150 mg every 12 hours (q12h) for 14 days. |
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| Part C: VX-121 (Cohort C2) | Experimental | Participants received VX-121 20 mg qd/TEZ 100 mg qd/IVA 150 mg q12h for 14 days. |
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| Part C: VX-121 (Cohort C3) | Experimental | Participants received VX-121 5 mg qd/TEZ 100 mg qd/IVA 150 mg q12h for 14 days. |
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| Part D: Placebo | Placebo Comparator | Participants received placebo matched to VX-121/TEZ/IVA for 4 weeks. |
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| Part D: VX-121/TEZ/IVA | Experimental | Participants received VX-121 5 mg qd/TEZ 100 mg qd/IVA 150 mg q12h for 4 weeks. |
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| VX-121 (Suspension) | Drug | Suspension for oral administration. |
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| TEZ/IVA | Drug | Fixed-dose combination tablet for oral administration. |
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| IVA | Drug | Tablet for oral administration. |
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| Placebo (matched to TEZ/IVA) | Drug | Placebo matched to TEZ/IVA for oral administration. |
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| Placebo (matched to IVA) | Drug | Placebo matched to IVA for oral administration. |
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| VX-121 (Tablet) | Drug | Tablet for oral administration. |
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| Placebo (matched to VX-121 tablet) | Drug | Placebo matched to VX-121 tablet for oral administration. |
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| Cohorts A1-5 (Except A3): Pre-dose up to 240 hours post-dose; Cohorts A3 and A9: Pre-dose up to 168 hours post-dose |
| Part B: Maximum Observed Concentration (Cmax) of VX-121 | Day 1, Day 5, and Day 10 |
| Part B: Area Under the Concentration Versus Time Curve During the Dosing Interval (AUCtau) of VX-121 | Day 1, Day 5, and Day 10 |
| Part B: Observed Pre-dose Plasma Concentration (Ctrough) of VX-121 | Pre-dose at Day 5 and Day 10 |
| Part C: Maximum Observed Concentration (Cmax) of VX-121, TEZ and Its Metabolites (M1-TEZ and M2-TEZ) and, IVA and Its Metabolites (M1-IVA and M6-IVA) | Day 1, Day 7, and Day 14 |
| Part C: Area Under the Concentration Versus Time Curve During a Dosing Interval (AUCtau) of VX-121, TEZ and Its Metabolites (M1-TEZ and M2-TEZ) and IVA and Its Metabolites (M1-IVA and M6-IVA) | Day 1, Day 7, and Day 14 |
| Part C: Pre-dose Plasma Concentration (Ctrough) of VX-121, TEZ and Its Metabolites (M1-TEZ and M2-TEZ) and IVA and Its Metabolites (M1-IVA and M6-IVA) | Pre-dose at Day 7 and Day 14 |
| Part D: Maximum Observed Concentration (Cmax) of VX-121, TEZ and Its Metabolites (M1-TEZ and M2-TEZ) and IVA and Its Metabolites (M1-IVA and M6-IVA) | Day 1 and Day 15 |
| Part D: Area Under the Concentration Versus Time Curve From the Time of Dosing to the Last Measurable Concentration (AUC[0-last]) of VX-121, TEZ and Its Metabolites (M1-TEZ and M2-TEZ) and IVA and Its Metabolites (M1-IVA and M6-IVA) | Day 1 and Day 15 |
| Part D: Pre-dose Plasma Concentration (Ctrough) of VX-121, TEZ and Its Metabolites (M1-TEZ and M2-TEZ) and IVA and Its Metabolites (M1-IVA and M6-IVA) | Pre-dose at Day 8, Day 15, and Day 29 |
| Part D: Absolute Change in Sweat Chloride (SwCl) Concentrations | Sweat samples were collected using an approved collection device. | From Baseline Through Day 29 |
| Part D: Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) | FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. | From Baseline Through Day 29 |
| Groningen |
| Netherlands |
| UMC St. Radboud | Nijmegen | Netherlands |
| Erasmus Medical Center | Rotterdam | Netherlands |
| HagaZiekenhuis van den Haag | The Hague | Netherlands |
| Heart of England NHS Foundation Trust, Birmingham Heartlands Hospital | Birmingham | United Kingdom |
| The Medicines Evaluation Unit | Manchester | United Kingdom |
| FG002 | Part A: VX-121 (Cohort A2) | Participants received single dose of VX-121 20 mg. |
| FG003 | Part A: VX-121 (Cohort A3) | Participants received single dose of VX-121 5 mg or matched placebo without milk, followed by open label VX-121 5 mg with milk. |
| FG004 | Part A: VX-121 (Cohort A4) | Participants received single dose of VX-121 40 mg. |
| FG005 | Part A: VX-121 (Cohort A5) | Participants received single dose of VX-121 60 mg. |
| FG006 | Part A: VX-121 (Cohort A9) | Participants received single dose of VX-121 10 mg suspension on Day 1, VX-121 10 mg tablet on Day 9, followed by VX-121 10 mg tablet with milk on Day 17. |
| FG007 | Part B: Pooled Placebo (Cohorts B1-4) | Participants received placebo matched to VX-121 for 10 days. |
| FG008 | Part B: VX-121 (Cohort B1) | Participants received VX-121 10 mg once daily (qd) for 10 days. |
| FG009 | Part B: VX-121 (Cohort B2) | Participants received VX-121 20 mg qd for 10 days. |
| FG010 | Part B: VX-121 (Cohort B3) | Participants received VX-121 40 mg qd for 10 days. |
| FG011 | Part B: VX-121 (Cohort B4) | Participants received VX-121 60 mg qd for 10 days. |
| FG012 | Part C: Pooled Placebo (Cohorts C1-3) | Participants received placebo matched to VX-121/TEZ/IVA for 14 days. |
| FG013 | Part C: VX-121 (Cohort C1) | Participants received VX-121 10 mg qd/TEZ 100 mg qd/IVA 150 mg every 12 hours (q12h) for 14 days. |
| FG014 | Part C: VX-121 (Cohort C2) | Participants received VX-121 20 mg qd/TEZ 100 mg qd/IVA 150 mg q12h for 14 days. |
| FG015 | Part C: VX-121 (Cohort C3) | Participants received VX-121 5 mg qd/TEZ 100 mg qd/IVA 150 mg q12h for 14 days. |
| FG016 | Part D: Placebo | Participants received placebo matched to VX-121/TEZ/IVA for 4 weeks. |
| FG017 | Part D: VX-121/TEZ/IVA | Participants received VX-121 5 mg qd/TEZ 100 mg qd/IVA 150 mg q12h for 4 weeks. |
| Safety Set | Received at least 1 dose of study drug. |
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| COMPLETED |
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| NOT COMPLETED |
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| Part B |
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| Part C |
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| Part D |
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Analysis population included all participants who received at least 1 dose of study drug. The participant who participated in Parts A2 and B3 counted only once in Part A: VX-121 (Cohort A2) reporting arm.
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| ID | Title | Description |
|---|---|---|
| BG000 | Part A: Pooled Placebo (Cohorts A1-5; Except A3) | Participants received single dose of placebo matched to VX-121. |
| BG001 | Part A: VX-121 (Cohort A1) | Participants received single dose of VX-121 10 mg. |
| BG002 | Part A: VX-121 (Cohort A2) | Participants received single dose of VX-121 20 mg. |
| BG003 | Part A: VX-121 (Cohort A3) | Participants received single dose of VX-121 5 mg or matched placebo without milk, followed by open label VX-121 5 mg with milk. |
| BG004 | Part A: VX-121 (Cohort A4) | Participants received single dose of VX-121 40 mg. |
| BG005 | Part A: VX-121 (Cohort A5) | Participants received single dose of VX-121 60 mg. |
| BG006 | Part A: Cohort A9 | Participants received single dose of VX-121 10 mg suspension on Day 1, VX-121 10 mg tablet on Day 9, followed by VX-121 10 mg tablet with milk on Day 17. |
| BG007 | Part B: Pooled Placebo (Cohorts B1-4) | Participants received placebo matched to VX-121 for 10 days. |
| BG008 | Part B: VX-121 (Cohort B1) | Participants received VX-121 10 mg qd for 10 days. |
| BG009 | Part B: VX-121 (Cohort B2) | Participants received VX-121 20 mg qd for 10 days. |
| BG010 | Part B: VX-121 (Cohort B3) | Participants received VX-121 40 mg qd for 10 days. |
| BG011 | Part B: VX-121 (Cohort B4) | Participants received VX-121 60 mg qd for 10 days. |
| BG012 | Part C: Pooled Placebo (Cohorts C1-3) | Participants received placebo matched to VX-121/TEZ/IVA in for 14 days. |
| BG013 | Part C: VX-121 (Cohort C1) | Participants received VX-121 10 mg qd/TEZ 100 mg qd/IVA 150 mg q12h for 14 days. |
| BG014 | Part C: VX-121 (Cohort C2) | Participants received VX-121 20 mg qd/TEZ 100 mg qd/IVA 150 mg q12h for 14 days. |
| BG015 | Part C: VX-121 (Cohort C3) | Participants received VX-121 5 mg qd/TEZ 100 mg qd/IVA 150 mg q12h for 14 days. |
| BG016 | Part D: Placebo | Participants received placebo matched to VX-121/TEZ/IVA for 4 weeks. |
| BG017 | Part D: VX-121/TEZ/IVA | Participants received VX-121 5 mg qd/TEZ 100 mg qd/IVA 150 mg q12h for 4 weeks. |
| BG018 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
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| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants | Participants |
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| Sex: Female, Male | Count of Participants | Participants |
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| Ethnicity (NIH/OMB) | Count of Participants | Participants |
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| Race (NIH/OMB) | Count of Participants | Participants |
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| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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| Primary | Safety and Tolerability as Assessed by Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) | Safety Set included all participants who received at least 1 dose of study drug. | Posted | Number | participants | From Day 1 Through Safety Follow-up (up to Day 15 for Part A [except Cohorts A3 and A9], up to Day 26 for Cohort A3, up to Day 34 for Cohort A9, up to Day 20 for Part B, up to Day 24 for Part C and up to Week 9 for Part D) |
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| Secondary | Part A: Maximum Observed Concentration (Cmax) of VX-121 | The pharmacokinetic (PK) set included all participants who received at least 1 dose of study drug and for whom the primary PK data are considered sufficient and interpretable. | Posted | Mean | Standard Deviation | microgram per milliliter (mcg/mL) | Cohorts A1-5 (Except A3): Pre-dose up to 240 hours post-dose; Cohorts A3 and A9: Pre-dose up to 168 hours post-dose |
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| Secondary | Part A: Area Under the Concentration Versus Time Curve From the Time of Dosing to the Last Measurable Concentration (AUC[0-last]) of VX-121 | PK set. | Posted | Mean | Standard Deviation | microgram*hour per milliliter (mcg*h/mL) | Cohorts A1-5 (Except A3): Pre-dose up to 240 hours post-dose; Cohorts A3 and A9: Pre-dose up to 168 hours post-dose |
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| Secondary | Part B: Maximum Observed Concentration (Cmax) of VX-121 | PK set. Here, the "number analyzed" signifies those participants who were evaluable for this outcome measure at specified time points. | Posted | Mean | Standard Deviation | mcg/mL | Day 1, Day 5, and Day 10 |
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| Secondary | Part B: Area Under the Concentration Versus Time Curve During the Dosing Interval (AUCtau) of VX-121 | PK set. Here, the "number analyzed" signifies those participants who were evaluable for this outcome measure at specified time points. | Posted | Mean | Standard Deviation | mcg*h/mL | Day 1, Day 5, and Day 10 |
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| Secondary | Part B: Observed Pre-dose Plasma Concentration (Ctrough) of VX-121 | PK set. Here, the "number analyzed" signifies those participants who were evaluable for this outcome measure at specified time points. | Posted | Mean | Standard Deviation | mcg/mL | Pre-dose at Day 5 and Day 10 |
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| Secondary | Part C: Maximum Observed Concentration (Cmax) of VX-121, TEZ and Its Metabolites (M1-TEZ and M2-TEZ) and, IVA and Its Metabolites (M1-IVA and M6-IVA) | PK set. Here, the "number analyzed" signifies those participants who were evaluable for this outcome measure at specified time points. | Posted | Mean | Standard Deviation | mcg/mL | Day 1, Day 7, and Day 14 |
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| Secondary | Part C: Area Under the Concentration Versus Time Curve During a Dosing Interval (AUCtau) of VX-121, TEZ and Its Metabolites (M1-TEZ and M2-TEZ) and IVA and Its Metabolites (M1-IVA and M6-IVA) | PK set. Here, the "number analyzed" signifies those participants who were evaluable for this outcome measure at specified time points. | Posted | Mean | Standard Deviation | mcg*h/mL | Day 1, Day 7, and Day 14 |
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| Secondary | Part C: Pre-dose Plasma Concentration (Ctrough) of VX-121, TEZ and Its Metabolites (M1-TEZ and M2-TEZ) and IVA and Its Metabolites (M1-IVA and M6-IVA) | PK set. Here, the "overall number of participants analyzed" signifies those participants who were evaluable for this outcome measure and "number analyzed" signifies those participants who were evaluable for this outcome measure at specified timepoints. | Posted | Mean | Standard Deviation | mcg/mL | Pre-dose at Day 7 and Day 14 |
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| Secondary | Part D: Maximum Observed Concentration (Cmax) of VX-121, TEZ and Its Metabolites (M1-TEZ and M2-TEZ) and IVA and Its Metabolites (M1-IVA and M6-IVA) | PK set. | Posted | Mean | Standard Deviation | mcg/mL | Day 1 and Day 15 |
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| Secondary | Part D: Area Under the Concentration Versus Time Curve From the Time of Dosing to the Last Measurable Concentration (AUC[0-last]) of VX-121, TEZ and Its Metabolites (M1-TEZ and M2-TEZ) and IVA and Its Metabolites (M1-IVA and M6-IVA) | PK set. | Posted | Mean | Standard Deviation | mcg*h/mL | Day 1 and Day 15 |
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| Secondary | Part D: Pre-dose Plasma Concentration (Ctrough) of VX-121, TEZ and Its Metabolites (M1-TEZ and M2-TEZ) and IVA and Its Metabolites (M1-IVA and M6-IVA) | PK set. | Posted | Mean | Standard Deviation | mcg/mL | Pre-dose at Day 8, Day 15, and Day 29 |
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| Secondary | Part D: Absolute Change in Sweat Chloride (SwCl) Concentrations | Sweat samples were collected using an approved collection device. | Full analysis set (FAS) included all randomized participants who carry the intended CF transmembrane conductance regulator protein (CFTR) allele mutation and have received at least 1 dose of study drug in the treatment period. | Posted | Least Squares Mean | 95% Confidence Interval | millimole per liter (mmol/L) | From Baseline Through Day 29 |
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| Secondary | Part D: Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) | FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. | FAS. | Posted | Least Squares Mean | 95% Confidence Interval | percentage points | From Baseline Through Day 29 |
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From Day 1 Through Safety Follow-up (up to Day 15 for Part A [except Cohorts A3 and A9], up to Day 26 for Cohort A3, up to Day 34 for Cohort A9, up to Day 20 for Part B, up to Day 24 for Part C and up to Week 9 for Part D)
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Part A: Pooled Placebo (Cohorts A1-5; Except A3) | Participants received single dose of placebo matched to VX-121. | 0 | 8 | 0 | 8 | 1 | 8 |
| EG001 | Part A: VX-121 (Cohort A1) | Participants received single dose of VX-121 10 mg. | 0 | 5 | 0 | 5 | 4 | 5 |
| EG002 | Part A: VX-121 (Cohort A2) | Participants received single dose of VX-121 20 mg. | 0 | 6 | 0 | 6 | 2 | 6 |
| EG003 | Part A: VX-121 (Cohort A3) | Participants received single dose of VX-121 5 mg or matched placebo without milk, followed by open label VX-121 5 mg with milk. | 0 | 6 | 0 | 6 | 4 | 6 |
| EG004 | Part A: VX-121 (Cohort A4) | Participants received single dose of VX-121 40 mg. | 0 | 6 | 0 | 6 | 1 | 6 |
| EG005 | Part A: VX-121 (Cohort A5) | Participants received single dose of VX-121 60 mg. | 0 | 6 | 0 | 6 | 2 | 6 |
| EG006 | Part A: VX-121 (Cohort A9) | Participants received single dose of VX-121 10 mg suspension on Day 1, VX-121 10 mg tablet on Day 9, followed by VX-121 10 mg tablet with milk on Day 17. | 0 | 8 | 0 | 8 | 7 | 8 |
| EG007 | Part B: Pooled Placebo (Cohorts B1-4) | Participants received placebo matched to VX-121 for 10 days. | 0 | 9 | 0 | 9 | 7 | 9 |
| EG008 | Part B: VX-121 (Cohort B1) | Participants received VX-121 10 mg qd for 10 days. | 0 | 6 | 0 | 6 | 3 | 6 |
| EG009 | Part B: VX-121 (Cohort B2) | Participants received VX-121 20 mg qd for 10 days. | 0 | 6 | 0 | 6 | 5 | 6 |
| EG010 | Part B: VX-121 (Cohort B3) | Participants received VX-121 40 mg qd for 10 days. | 0 | 6 | 0 | 6 | 4 | 6 |
| EG011 | Part B: VX-121 (Cohort B4) | Participants received VX-121 60 mg qd for 10 days. | 0 | 6 | 0 | 6 | 5 | 6 |
| EG012 | Part C: Pooled Placebo (Cohorts C1-3) | Participants received placebo matched to VX-121/TEZ/IVA for 14 days. | 0 | 6 | 0 | 6 | 4 | 6 |
| EG013 | Part C: VX-121 (Cohort C1) | Participants received VX-121 10 mg qd/TEZ 100 mg qd/IVA 150 mg q12h for 14 days. | 0 | 6 | 0 | 6 | 3 | 6 |
| EG014 | Part C: VX-121 (Cohort C2) | Participants received VX-121 20 mg qd/TEZ 100 mg qd/IVA 150 mg q12h for 14 days. | 0 | 7 | 0 | 7 | 6 | 7 |
| EG015 | Part C: VX-121 (Cohort C3) | Participants received VX-121 5 mg qd/TEZ 100 mg qd/IVA 150 mg q12h for 14 days. | 0 | 6 | 0 | 6 | 3 | 6 |
| EG016 | Part D: Placebo | Participants received placebo matched to VX-121/TEZ/IVA for 4 weeks. | 0 | 3 | 1 | 3 | 2 | 3 |
| EG017 | Part D: VX-121/TEZ/IVA | Participants received VX-121 5 mg qd/TEZ 100 mg qd/IVA 150 mg q12h for 4 weeks. | 0 | 9 | 0 | 9 | 7 | 9 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Infective pulmonary exacerbation of cystic fibrosis | Infections and infestations | MedDRA 22.0 | Systematic Assessment |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Lymphadenopathy | Blood and lymphatic system disorders | MedDRA 22.0 | Systematic Assessment |
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| Ear pain | Ear and labyrinth disorders | MedDRA 22.0 | Systematic Assessment |
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| Eye irritation | Eye disorders | MedDRA 22.0 | Systematic Assessment |
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| Ocular hyperaemia | Eye disorders | MedDRA 22.0 | Systematic Assessment |
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| Abdominal distension | Gastrointestinal disorders | MedDRA 22.0 | Systematic Assessment |
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| Abdominal pain | Gastrointestinal disorders | MedDRA 22.0 | Systematic Assessment |
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| Abdominal pain upper | Gastrointestinal disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Defaecation urgency | Gastrointestinal disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Dyspepsia | Gastrointestinal disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Flatulence | Gastrointestinal disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Oral pain | Gastrointestinal disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Salivary hypersecretion | Gastrointestinal disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Toothache | Gastrointestinal disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Asthenia | General disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Catheter site bruise | General disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Catheter site erythema | General disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Catheter site irritation | General disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Catheter site pain | General disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Catheter site pruritus | General disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Chest discomfort | General disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Fatigue | General disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Feeling cold | General disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Feeling hot | General disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Injection site bruising | General disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Puncture site pain | General disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Vessel puncture site bruise | General disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Vessel puncture site pain | General disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Infective pulmonary exacerbation of cystic fibrosis | Infections and infestations | MedDRA 22.0 | Systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA 22.0 | Systematic Assessment |
| |
| Periodontitis | Infections and infestations | MedDRA 22.0 | Systematic Assessment |
| |
| Arthropod bite | Injury, poisoning and procedural complications | MedDRA 22.0 | Systematic Assessment |
| |
| Contusion | Injury, poisoning and procedural complications | MedDRA 22.0 | Systematic Assessment |
| |
| Procedural complication | Injury, poisoning and procedural complications | MedDRA 22.0 | Systematic Assessment |
| |
| Blood creatine phosphokinase increased | Investigations | MedDRA 22.0 | Systematic Assessment |
| |
| Faecal volume decreased | Investigations | MedDRA 22.0 | Systematic Assessment |
| |
| Influenza A virus test positive | Investigations | MedDRA 22.0 | Systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Flank pain | Musculoskeletal and connective tissue disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Muscle twitching | Musculoskeletal and connective tissue disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Musculoskeletal chest pain | Musculoskeletal and connective tissue disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Myalgia | Musculoskeletal and connective tissue disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Dizziness | Nervous system disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Dizziness postural | Nervous system disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Nerve compression | Nervous system disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Ophthalmoplegic migraine | Nervous system disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Somnolence | Nervous system disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Syncope | Nervous system disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Anxiety | Psychiatric disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Insomnia | Psychiatric disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Restlessness | Psychiatric disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Tension | Psychiatric disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Pollakiuria | Renal and urinary disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Nasal congestion | Respiratory, thoracic and mediastinal disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Painful respiration | Respiratory, thoracic and mediastinal disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Productive cough | Respiratory, thoracic and mediastinal disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Rhinorrhoea | Respiratory, thoracic and mediastinal disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Sputum increased | Respiratory, thoracic and mediastinal disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Alopecia | Skin and subcutaneous tissue disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Blister | Skin and subcutaneous tissue disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Dermal cyst | Skin and subcutaneous tissue disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Dry skin | Skin and subcutaneous tissue disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Erythema | Skin and subcutaneous tissue disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Hyperhidrosis | Skin and subcutaneous tissue disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Night sweats | Skin and subcutaneous tissue disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Papule | Skin and subcutaneous tissue disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Pruritus | Skin and subcutaneous tissue disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Pruritus generalised | Skin and subcutaneous tissue disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Rash generalised | Skin and subcutaneous tissue disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Rash macular | Skin and subcutaneous tissue disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Rash maculo-papular | Skin and subcutaneous tissue disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Seborrhoeic dermatitis | Skin and subcutaneous tissue disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Skin hypertrophy | Skin and subcutaneous tissue disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Skin hypopigmentation | Skin and subcutaneous tissue disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Skin irritation | Skin and subcutaneous tissue disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Swelling face | Skin and subcutaneous tissue disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Phlebitis | Vascular disorders | MedDRA 22.0 | Systematic Assessment |
|
Not provided
Not provided
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Medical Monitor | Vertex Pharmaceuticals Incorporated | 617-341-6777 | medicalinfo@vrtx.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Aug 28, 2018 | Apr 29, 2022 | SAP_001.pdf |
Not provided
| ID | Term |
|---|---|
| D003550 | Cystic Fibrosis |
| ID | Term |
|---|---|
| D010182 | Pancreatic Diseases |
| D004066 | Digestive System Diseases |
| D008171 | Lung Diseases |
| D012140 | Respiratory Tract Diseases |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D007232 | Infant, Newborn, Diseases |
Not provided
Not provided
| ID | Term |
|---|---|
| D013535 | Suspensions |
| C000654124 | tezacaftor, ivacaftor drug combination |
| C545203 | ivacaftor |
| D013607 | Tablets |
| ID | Term |
|---|---|
| D003102 | Colloids |
| D045424 | Complex Mixtures |
| D004304 | Dosage Forms |
| D004364 | Pharmaceutical Preparations |
Not provided
Not provided
| Between 18 and 65 years |
|
| >=65 years |
|
| Male |
|
| Not Hispanic or Latino |
|
| Unknown or Not Reported |
|
| Asian |
|
| Native Hawaiian or Other Pacific Islander |
|
| Black or African American |
|
| White |
|
| More than one race |
|
| Unknown or Not Reported |
|
| Participants With SAEs |
|
Participants received single dose of VX-121 40 mg. |
| OG005 | Part A: VX-121 (Cohort A5) | Participants received single dose of VX-121 60 mg. |
| OG006 | Part A: VX-121 (Cohort A9: Suspension) | Participants received single dose of VX-121 10 mg suspension on Day 1. |
| OG007 | Part A: VX-121 (Cohort A9: Tablet Without Milk) | Participants received single dose of VX-121 10 mg tablet without milk on Day 9. |
| OG008 | Part A: VX-121 (Cohort A9: Tablet With Milk) | Participants received single dose of VX-121 10 mg tablet with milk on Day 17. |
|
|
Participants received single dose of VX-121 40 mg.
| OG005 | Part A: VX-121 (Cohort A5) | Participants received single dose of VX-121 60 mg. |
| OG006 | Part A: VX-121 (Cohort A9: Suspension) | Participants received single dose of VX-121 10 mg suspension on Day 1. |
| OG007 | Part A: VX-121 (Cohort A9: Tablet Without Milk) | Participants received single dose of VX-121 10 mg tablet without milk on Day 9. |
| OG008 | Part A: VX-121 (Cohort A9: Tablet With Milk) | Participants received single dose of VX-121 10 mg tablet with milk on Day 17. |
|
|
|
|
| Participants |
|
|
|
|
|
|
|
|
| Denominators |
|---|
| Categories |
|---|
| VX-121: Day 1 |
| |||||
| VX-121: Day 15 |
| |||||
| TEZ: Day 1 |
| |||||
| TEZ: Day 15 |
| |||||
| M1-TEZ: Day 1 |
| |||||
| M1-TEZ: Day 15 |
| |||||
| M2-TEZ: Day 1 |
| |||||
| M2-TEZ: Day 15 |
| |||||
| IVA: Day 1 |
| |||||
| IVA: Day 15 |
| |||||
| M1-IVA: Day 1 |
| |||||
| M1-IVA: Day 15 |
| |||||
| M6-IVA: Day 1 |
| |||||
| M6-IVA: Day 15 |
|
| Title | Denominators | Categories |
|---|
| VX-121: Day 1 |
| |||||
| VX-121: Day 15 |
| |||||
| TEZ: Day 1 |
| |||||
| TEZ: Day 15 |
| |||||
| M1-TEZ: Day 1 |
| |||||
| M1-TEZ: Day 15 |
| |||||
| M2-TEZ: Day 1 |
| |||||
| M2-TEZ: Day 15 |
| |||||
| IVA: Day 1 |
| |||||
| IVA: Day 15 |
| |||||
| M1-IVA: Day 1 |
| |||||
| M1-IVA: Day 15 |
| |||||
| M6-IVA: Day 1 |
| |||||
| M6-IVA: Day 15 |
|
| Title |
|---|
| Denominators |
|---|
| Categories |
|---|
| VX-121: Day 8 |
| |||||
| VX-121: Day 15 |
| |||||
| VX-121: Day 29 |
| |||||
| TEZ: Day 8 |
| |||||
| TEZ: Day 15 |
| |||||
| TEZ: Day 29 |
| |||||
| M1-TEZ: Day 8 |
| |||||
| M1-TEZ: Day 15 |
| |||||
| M1-TEZ: Day 29 |
| |||||
| M2-TEZ: Day 8 |
| |||||
| M2-TEZ: Day 15 |
| |||||
| M2-TEZ: Day 29 |
| |||||
| IVA: Day 8 |
| |||||
| IVA: Day 15 |
| |||||
| IVA: Day 29 |
| |||||
| M1-IVA: Day 8 |
| |||||
| M1-IVA: Day 15 |
| |||||
| M1-IVA: Day 29 |
| |||||
| M6-IVA: Day 8 |
| |||||
| M6-IVA: Day 15 |
| |||||
| M6-IVA: Day 29 |
|
|
|
| Title | Measurements |
|---|---|
|
| Title | Measurements |
|---|---|
|
| Title | Measurements |
|---|---|
|
| Title | Measurements |
|---|---|
|
| Title | Measurements |
|---|---|
|